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Pediatric cancers can now boast a survival rate of better than 80 percent. A major reason for this success is enrollment of patients onto clinical trials. Clinical trials in pediatric cancers are rationally designed to deliver the most effective therapy known in addition to testing new combinations or medications that can lead to an even better cure rate. Such innovation is vital not only to the sustained success of treating children with cancer but is also a major focus here.
History of Pioneering Treatments
Memorial Sloan-Kettering has a long history of innovative therapies and treatment approaches for pediatric sarcomas. Clinical trials first conducted here, and later emulated by other cancer centers nationally, have led to the development of extremely effective treatments for patients with sarcomas. For example, in the 1950s, patients with osteosarcoma had an overall survival rate of less than 15 percent compared with the better than 80 percent overall survival rate we've achieved today.
Our physicians were among the first to recognize the importance of systemic chemotherapy in this disease. This recognition eventually led to the development of protocols that have since resulted in an 80 percent survival rate among patients with non-metastatic osteosarcoma. Many of the effective chemotherapy agents and combinations of chemotherapy agents in use today were first used in clinical trials here.
Physicians on Memorial Sloan-Kettering's Pediatric Sarcoma Team continue to lead many of the national clinical trials to improve treatment outcomes for these diseases across the country. Our physicians have chaired the Bone Tumor Strategy Group of the Children's Cancer Group, a national cooperative group for centers specializing in pediatric cancers. And our specialists continue to head efforts, both here at Memorial Sloan-Kettering as well as in the national cooperative groups, to improve cure rates in pediatric sarcomas.
Memorial Sloan-Kettering staff have led the national cooperative group's laboratory research efforts and have identified crucial prognostic factors in osteosarcoma; explained some of the mechanisms by which osteosarcoma tumor cells develop resistance to chemotherapy; and developed novel therapies to overcome this resistance. Our physicians are also actively involved in developing new therapeutic strategies for soft tissue sarcomas both here at Memorial Sloan-Kettering as well as in the national cooperative groups.
Patient-Tailored Therapies
Going forward, our researchers will continue to develop new therapies and surgical techniques. The new therapies being developed for pediatric sarcomas focus on delivering effective tumor killing agents while reducing the side-effects associated with therapy. These innovations are designed to tailor cancer therapies specific to each individual patient's metabolism and extent of disease.
For example, after promising laboratory studies with trimetrexate (a drug related to methotrexate, a standard chemotherapy agent used to treat osteosarcoma), doctors at Memorial Sloan-Kettering have begun important clinical studies of the drug for the treatment of recurrent osteosarcoma. In addition, we are testing the effectiveness of inhaled cisplatin (its intravenous formulation is a treatment standard for osteosarcoma) in the treatment of patients with relapsed osteosarcoma confined to the lungs. This exciting new approach effectively delivers chemotherapy to sites of tumor involvement without affecting the rest of the body, thus reducing the potential side-effects of chemotherapy.
Immune System Stimulation & High-Dose Chemotherapy
For patients with Ewing's sarcoma, we are testing to see if stimulation of the immune system in addition to chemotherapy will help achieve a cure in Ewing's sarcoma. In an effort to improve outcomes in certain high-risk sarcomas, we are evaluating the effectiveness of high-dose chemotherapy followed by allogeneic bone marrow transplantation (in which bone marrow from a patient's own family is given to the patient after a high-dose chemotherapy regimen) in patients with high-risk sarcomas.